Theratechnologies Inc. came up with disappointing results from a Phase II trial with its stabilized analogue of the growth hormone-releasing factor for muscle wasting related to hip fracture surgery, but the company said development of the drug in other indications will forge ahead.
"This is one of three irons we have in the fire," said Peter McBride, vice president of investor relations and public affairs for Montreal-based Theratechnologies, noting that the drug also is being studied against wasting in chronic obstructive pulmonary disease (COPD) patients and against HIV-related lipodystrophy.
Positive early Phase II data from the COPD trial were disclosed in October, and results from the study in lipodystrophy - which is "the [indication] analysts like best," McBride said - are due at the end of March.
The market took the hip-fracture news hard, denting the company's shares (TSX:TH) Tuesday by 25.8 percent, dropping it by C$1.17 to close at C$3.37 (US$2.58).
In the hip-fracture study, ThGRF offered the same good safety profile of earlier studies but failed to bring improvement in functional recovery after hip-fracture surgery among elderly patients, according to the analysis of preliminary data from two clinically important parameters: the Lower Extremity Measure, and the Functional Autonomy Measurement System.
"These people are, in fact, in too much of a distress state to help them," McBride said. "You have to allow them to stabilize and maybe intervene in two or three months."
Conducted at 20 centers in Canada and Europe, the study was a double-blind, randomized, parallel-group and placebo-controlled effort. A dose of 2 mg or placebo was administered daily to 127 patients who were 80 years old on average, for a period of eight weeks, followed by evaluation one month after treatment.
Functional recovery was measured based on criteria that prove the ability to perform daily activities and that gauge autonomy.
"In COPD, we were able to induce some very important changes in body composition," McBride said. "In this case, we weren't able to do anything."
ThGRF, which is licensed in Japan to Sakai Chemical Industry Ltd., of Tokyo, had only a weak effect on insulin-like growth factor 1 (IGF-1) in the tested patients. IGF-1 levels on average rose only 28 percent over placebo. In the COPD trial, the same dose boosted IGF-1 levels an average of 100 percent.
McBride said the company might revisit the hip-fracture indication later, but immediate efforts are focused on devising a later-stage COPD trial with the FDA, while awaiting the lipodystrophy data.
Another possibility for ThGRF is in pediatric indications.
"We have not been pursuing that very aggressively, although we have a program ongoing," McBride told BioWorld Today. "It's a well-served market already."
Instead, the company is making the most of ThGRF's main advantage.
"Our product doesn't have the safety concerns that recombinant growth hormone has in adult populations," he said, one of the more troublesome of which is the risk of diabetes. "We specifically did a safety trial in older diabetics and came up very clean."